Zinc Finger Nucleases: Tailor-made for Gene Therapy.

PubWeight™: 0.79‹?›

🔗 View Article (PMID 24155503)

Published in Drugs Future on March 01, 2012

Authors

S-T Chou1, Qixin Leng, A J Mixson

Author Affiliations

1: Department of Pathology, University of Maryland School of Medicine, MSTF Building, 10 South Pine Street, Baltimore, MD 21201, USA ; Department of Chemical and Biomolecular Engineering, University of Maryland, College Park, MD 20742.

Associated clinical trials:

Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 for HIV (Zinc-Finger) | NCT00842634

Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients | NCT01044654

Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects | NCT01252641

Phase I Study of Cellular Immunotherapy for Recurrent/Refractory Malignant Glioma Using Intratumoral Infusions of GRm13Z40-2, An Allogeneic CD8+ Cytolitic T-Cell Line Genetically Modified to Express the IL 13-Zetakine and HyTK and to be Resistant to Glucocorticoids, in Combination With Interleukin-2 | NCT01082926

Articles cited by this

(truncated to the top 100)

Repetitive zinc-binding domains in the protein transcription factor IIIA from Xenopus oocytes. EMBO J (1985) 20.39

Multiple pathways of recombination induced by double-strand breaks in Saccharomyces cerevisiae. Microbiol Mol Biol Rev (1999) 19.25

Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature (2005) 19.15

Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88

An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol (2007) 17.80

Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 A. Science (1991) 15.97

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol (2008) 14.75

Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat Biotechnol (2008) 14.20

Altering the genome by homologous recombination. Science (1989) 12.73

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol (2009) 12.40

Knockout rats via embryo microinjection of zinc-finger nucleases. Science (2009) 12.33

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A (1996) 12.25

Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. Mol Cell (2008) 11.24

Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases. Nat Biotechnol (2008) 10.29

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23

Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics (2002) 9.09

Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cell (2009) 8.97

Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA). Nat Methods (2010) 8.86

Distinct factors control histone variant H3.3 localization at specific genomic regions. Cell (2010) 8.79

Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly. Genome Res (2009) 8.69

Role of RAD52 epistasis group genes in homologous recombination and double-strand break repair. Microbiol Mol Biol Rev (2002) 8.50

Enhancing gene targeting with designed zinc finger nucleases. Science (2003) 8.42

High-frequency modification of plant genes using engineered zinc-finger nucleases. Nature (2009) 7.19

Molecular views of recombination proteins and their control. Nat Rev Mol Cell Biol (2003) 6.96

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Nat Methods (2011) 6.90

Chromosomal stability and the DNA double-stranded break connection. Nat Rev Genet (2001) 6.77

Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol (2001) 6.68

Rapid mutation of endogenous zebrafish genes using zinc finger nucleases made by Oligomerized Pool ENgineering (OPEN). PLoS One (2009) 6.67

Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci U S A (2008) 6.59

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55

An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44

Targeted chromosomal deletions in human cells using zinc finger nucleases. Genome Res (2009) 6.24

Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells. Proc Natl Acad Sci U S A (2006) 6.00

Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci U S A (2007) 5.93

Zinc finger phage: affinity selection of fingers with new DNA-binding specificities. Science (1994) 5.70

Zinc-finger nucleases: the next generation emerges. Mol Ther (2008) 5.69

Targeted integration in rat and mouse embryos with zinc-finger nucleases. Nat Biotechnol (2010) 5.60

Genetic manipulation of genomes with rare-cutting endonucleases. Trends Genet (1996) 5.07

A general strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites. Science (1997) 4.93

Toward controlling gene expression at will: selection and design of zinc finger domains recognizing each of the 5'-GNN-3' DNA target sequences. Proc Natl Acad Sci U S A (1999) 4.87

In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81

Precise genome modification in the crop species Zea mays using zinc-finger nucleases. Nature (2009) 4.71

Efficient gene targeting in Drosophila with zinc-finger nucleases. Genetics (2006) 4.47

Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains. Nucleic Acids Res (2000) 4.34

Human zinc fingers as building blocks in the construction of artificial transcription factors. Nat Biotechnol (2003) 4.03

Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae. Mol Cell Biol (1995) 3.96

DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases. Mol Ther (2007) 3.94

Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases. Proc Natl Acad Sci U S A (2010) 3.90

Efficient gene targeting in Drosophila by direct embryo injection with zinc-finger nucleases. Proc Natl Acad Sci U S A (2008) 3.70

ZiFiT (Zinc Finger Targeter): an updated zinc finger engineering tool. Nucleic Acids Res (2010) 3.59

Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med (2010) 3.54

RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci Transl Med (2010) 3.53

Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res (2005) 3.47

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood (2011) 3.43

Technical challenges in using human induced pluripotent stem cells to model disease. Cell Stem Cell (2009) 3.42

Targeted genome modification in mice using zinc-finger nucleases. Genetics (2010) 3.38

DNA breakage and repair. Adv Genet (1998) 3.38

Development of zinc finger domains for recognition of the 5'-ANN-3' family of DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem (2001) 3.35

A bacterial two-hybrid selection system for studying protein-DNA and protein-protein interactions. Proc Natl Acad Sci U S A (2000) 3.35

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood (2011) 3.21

A rapid, generally applicable method to engineer zinc fingers illustrated by targeting the HIV-1 promoter. Nat Biotechnol (2001) 3.12

Generation of knockout rats with X-linked severe combined immunodeficiency (X-SCID) using zinc-finger nucleases. PLoS One (2010) 3.09

Custom zinc-finger nucleases for use in human cells. Mol Ther (2005) 3.04

Expanding or restricting the target site repertoire of zinc-finger nucleases: the inter-domain linker as a major determinant of target site selectivity. Mol Ther (2008) 3.02

Development of zinc finger domains for recognition of the 5'-CNN-3' family DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem (2005) 2.69

Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases. J Mol Biol (2010) 2.65

Mammalian gene targeting with designed zinc finger nucleases. Mol Ther (2005) 2.62

Efficient generation of a biallelic knockout in pigs using zinc-finger nucleases. Proc Natl Acad Sci U S A (2011) 2.61

Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. Nucleic Acids Res (2010) 2.61

Specific recognition and killing of glioblastoma multiforme by interleukin 13-zetakine redirected cytolytic T cells. Cancer Res (2004) 2.54

In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. Stem Cells (2011) 2.52

Determination of the base recognition positions of zinc fingers from sequence analysis. EMBO J (1992) 2.31

Genetic analysis of zinc-finger nuclease-induced gene targeting in Drosophila. Genetics (2009) 2.27

Adeno-associated virus vectors integrate at chromosome breakage sites. Nat Genet (2004) 2.27

Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases. Biotechnol Bioeng (2010) 2.23

Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels. PLoS Genet (2009) 2.18

Cytosine methylation targetted to pre-determined sequences. Nat Genet (1997) 1.81

Zinc finger protein-dependent and -independent contributions to the in vivo off-target activity of zinc finger nucleases. Nucleic Acids Res (2010) 1.81

Efficient oncogene silencing and metastasis inhibition via systemic delivery of siRNA. Mol Ther (2008) 1.74

Generation of PPARγ mono-allelic knockout pigs via zinc-finger nucleases and nuclear transfer cloning. Cell Res (2011) 1.74

Physicochemical and biological characterization of targeted, nucleic acid-containing nanoparticles. Bioconjug Chem (2007) 1.71

Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases. PLoS One (2011) 1.69

Site-directed mutagenesis in Arabidopsis using custom-designed zinc finger nucleases. Proc Natl Acad Sci U S A (2010) 1.65

Targeted mutagenesis in the sea urchin embryo using zinc-finger nucleases. Genes Cells (2010) 1.64

Zinc-finger nucleases as a novel therapeutic strategy for targeting hepatitis B virus DNAs. Mol Ther (2010) 1.61

Zinc-finger nuclease based genome surgery: it's all about specificity. Curr Gene Ther (2011) 1.56

Custom DNA-binding proteins come of age: polydactyl zinc-finger proteins. Curr Opin Biotechnol (2001) 1.52

Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases. PLoS Pathog (2011) 1.50

Characterization of anti-CCR5 ribozyme-transduced CD34+ hematopoietic progenitor cells in vitro and in a SCID-hu mouse model in vivo. Mol Ther (2000) 1.47

BAK and BAX deletion using zinc-finger nucleases yields apoptosis-resistant CHO cells. Biotechnol Bioeng (2010) 1.46

Traceable multifunctional micellar nanocarriers for cancer-targeted co-delivery of MDR-1 siRNA and doxorubicin. ACS Nano (2011) 1.46

Integrase-defective lentiviral vectors: progress and applications. Gene Ther (2009) 1.41

Computer-assisted hydrodynamic gene delivery. Mol Ther (2008) 1.40

Zinc finger nucleases as tools to understand and treat human diseases. BMC Med (2010) 1.36

I-SceI-induced gene replacement at a natural locus in embryonic stem cells. Mol Cell Biol (1998) 1.32

Potent suppression of HIV type 1 infection by a short hairpin anti-CXCR4 siRNA. AIDS Res Hum Retroviruses (2003) 1.23

Highly efficient modification of beta-lactoglobulin (BLG) gene via zinc-finger nucleases in cattle. Cell Res (2011) 1.23

Image-guided, lobe-specific hydrodynamic gene delivery to swine liver. Mol Ther (2009) 1.20

Bispecific short hairpin siRNA constructs targeted to CD4, CXCR4, and CCR5 confer HIV-1 resistance. Oligonucleotides (2003) 1.20

Adding fingers to an engineered zinc finger nuclease can reduce activity. Biochemistry (2011) 1.17

Articles by these authors

Attention deficit-hyperactivity disorder in people with generalized resistance to thyroid hormone. N Engl J Med (1993) 3.62

Characterization of seven novel mutations of the c-erbA beta gene in unrelated kindreds with generalized thyroid hormone resistance. Evidence for two "hot spot" regions of the ligand binding domain. J Clin Invest (1991) 2.40

ALDH1A1 is a marker for malignant prostate stem cells and predictor of prostate cancer patients' outcome. Lab Invest (2009) 2.25

Liposomes complexed to plasmids encoding angiostatin and endostatin inhibit breast cancer in nude mice. Cancer Res (1999) 2.08

Aldehyde dehydrogenase 1 A1-positive cell population is enriched in tumor-initiating cells and associated with progression of bladder cancer. Cancer Epidemiol Biomarkers Prev (2010) 1.85

EAPII interacts with ETS1 and modulates its transcriptional function. Oncogene (2003) 1.51

Parenteral gene therapy with p53 inhibits human breast tumors in vivo through a bystander mechanism without evidence of toxicity. Hum Gene Ther (1997) 1.11

Branched co-polymers of histidine and lysine are efficient carriers of plasmids. Nucleic Acids Res (2001) 1.04

Systemic gene therapy with p53 reduces growth and metastases of a malignant human breast cancer in nude mice. Hum Gene Ther (1995) 0.98

Peptide-based Antifungal Therapies against Emerging Infections. Drugs Future (2010) 0.97

Co-polymer of histidine and lysine markedly enhances transfection efficiency of liposomes. Gene Ther (2000) 0.96

Alteration in the IL-2 signal peptide affects secretion of proteins in vitro and in vivo. J Gene Med (2005) 0.93

Selective modification of HK peptides enhances siRNA silencing of tumor targets in vivo. Cancer Gene Ther (2011) 0.92

Synthetic histidine-rich peptides inhibit Candida species and other fungi in vitro: role of endocytosis and treatment implications. Antimicrob Agents Chemother (2006) 0.91

Systemic delivery of HK Raf-1 siRNA polyplexes inhibits MDA-MB-435 xenografts. Cancer Gene Ther (2008) 0.90

Enhanced silencing and stabilization of siRNA polyplexes by histidine-mediated hydrogen bonds. Biomaterials (2013) 0.86

Surface-modified HK:siRNA nanoplexes with enhanced pharmacokinetics and tumor growth inhibition. Biomacromolecules (2013) 0.86

Analysis of the binding of 3,3',5-triiodo-L-thyronine and its analogues to mutant human beta 1 thyroid hormone receptors: a model of the hormone binding site. Biochemistry (1994) 0.85

Targeting tumor angiogenesis with gene therapy. Mol Genet Metab (2001) 0.85

Regulation of differentiation by a PHD domain in the NUP98-PHF23 fusion protein. Leuk Res (2010) 0.84

Vaccines targeting the neovasculature of tumors. Vasc Cell (2011) 0.84

In vivo gene therapy with a cationic polymer markedly enhances the antitumor activity of antiangiogenic genes. Mol Genet Metab (1998) 0.79

Systemic gene therapy with p53 inhibits breast cancer: recent advances and therapeutic implications. Front Biosci (1998) 0.79

Gene therapy with p53 and a fragment of thrombospondin I inhibits human breast cancer in vivo. Mol Genet Metab (1998) 0.79

Enhancement of antifungal activity by integrin-targeting of branched histidine rich peptides. J Drug Target (2014) 0.78

Direct observation of dynamic mechanical regulation of DNA condensation by environmental stimuli. Angew Chem Int Ed Engl (2014) 0.76

ADHD and the thyroid controversy. J Am Acad Child Adolesc Psychiatry (1994) 0.75

The proto-oncogenes c-fos and c-jun modulate thyroid hormone inhibition of human thyrotropin beta subunit gene expression in opposite directions. Biochem Biophys Res Commun (1992) 0.75